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Biogen Idec is paying Isis Pharmaceuticals $29 million up front to seal an exclusive, worldwide option and collaboration agreement centered on development and commercialization of the latter’s Phase I-stage antisense candidate for the treatment of spinal muscular atrophy, ISIS-SMNRX. Under terms of the deal Biogen Idec retains an option to license ISIS-SMNRX until completion of the first successful Phase II/III study.

Isis could receive up to $45 million in milestone payments associated with clinical development of ISIS-SMNRX prior to licensing. If Biogen Idec takes up its option the firm could earn another $255 million in license fees and regulatory milestone payments plus double-digit sales royalties.

Isis will shoulder global development of ISIS-SMNRX through completion of Phase II/III registrational trials, with Biogen Idec providing advice on clinical trial design and regulatory strategy. If the latter exercises its option to license the drug, the firm will take over all global development, regulatory, and comemrcialization activities.

“Biogen Idec’s expertise in the global development and commercialization of innovative new therapies for neurologic diseases is a great strategic fit to advance ISIS-SMNRX," comments Stanley T. Crooke, M.D., CEO and board chairman at Isis. “This alliance is consistent with our business strategy to develop antisense drugs to proof-of-concept with a knowledgeable partner that is committed to supporting the rapid development of the drug.” Isis says the need to develop a treatment for SMA means proof-of-concept studies with ISIS-SMNRX should also suffice as registrational trials.

Isis is exploiting its antisense drug discovery platform to develop a broad pipeline of drugs against a range of diseases. SMA is caused by the loss of or defect in the SMN1 gene, which results in the lack, to varying degrees, of the SMN protein that is critical to nerve cell survival in the spinal cord. ISIS-SMNRX is designed to treat all types of the childhood SMA by altering splicing of the closely related SMN2 gene, resulting in increased production of fully functional SMN protein.

An initial Phase I trial evaluating ISIS-SMNRX in children with SMA was initiated just last month. The study is evaluating the safety, tolerability, and pharmacokinetics of a single, intrathecally administered injection of the drug into the spinal fluid of medically stable SMA patients aged 2 to 14 years.

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